Press Releases

12/6/2006

PHILADELPHIA, Dec. 6 /PRNewswire/ -- While survival rates for childhoodleukemia have dramatically improved over the past 30 years, infants withthis blood cancer continue to face difficult odds. Infant leukemia resiststreatments such as chemotherapy and stem cell transplants that may beeffective in older children, and infants are especially vulnerable totreatment side effects.

A new research effort led by a leukemia expert at The Children'sHospital of Philadelphia seeks to harness in-depth understanding of genesand molecular pathways to develop highly specific drugs designed to killleukemia cells while causing few or no toxic effects on normal cells.

A five-year, $6.25-million Specialized Center of Research (SCOR) grantfrom the Leukemia & Lymphoma Society brings together a diverse study teamof researchers from leading medical centers to develop innovativetreatments for infant leukemia. "Our goal is to streamline advances inmolecular medicine to find new treatment options," said Carolyn A. Felix,M.D., principal investigator of the SCOR grant. A pediatric oncologist atThe Children's Hospital of Philadelphia, Dr. Felix also is a professor ofPediatrics at the University of Pennsylvania School of Medicine.

The society recently announced its grant award in targeted therapiesfor infant leukemia from its national office in White Plains, N.Y. The SCORgrant draws on research collaborators from a variety of disciplines todiscover new approaches to treatment.

In addition to researchers from The Children's Hospital of Philadelphiaand the University of Pennsylvania, the grant includes collaborators fromthe University of New Mexico, Johns Hopkins University, Stanford Universityand Tulane University.

Especially integral to this effort to advance new treatments from benchto bedside, the investigators also will work closely with the Children'sOncology Group, the cooperative clinical research organization of pediatriccancer centers throughout North America. "Even the largest medical centerhas a relatively small number of patients with infant leukemia," said Dr.Felix. "Collaborating with the Children's Oncology Group allows us tocapture data about and treat the vast majority of infants throughout NorthAmerica who have acute leukemia."

The full grant encompasses four projects, each of which hones in on apiece of the infant leukemia puzzle. In infant leukemia, a gene calledmixed lineage leukemia (MLL) breaks and recombines with one of many partnergenes to form a translocation, an abnormal rearrangement within achromosome. Because MLL plays a critical role in blood cell development,the translocation causes the overproduction of defective white blood cells that are the hallmark of leukemia.

Dr. Felix has pioneered methods to identify and describe the featuresof MLL translocations. Her project within the SCOR grant will investigatehow potential drugs may trigger programmed cell death, called apoptosis, in leukemia cells in infants. Another project, led by Cheryl L. Willman, M.D.,of the University of New Mexico, will use microarrays (DNA chips) toidentify drugs that may selectively attack cells with MLL translocations.

The project also seeks to unravel the molecular circuitry in leukemia cellsin order to predict those infants most likely to benefit from specifictargeted drugs.

Donald Small, M.D., Ph.D., of Johns Hopkins University, leads a SCORproject to advance the development of new drugs to target abnormalities ina gene called FLT3 that are associated with uncontrolled growth of theleukemia cell population.

A final team under the SCOR grant, led by Michael Cleary, M.D., ofStanford University, will characterize the MLL leukemia stem cell thatgives rise to all other leukemia cells, because the stem cells areessential targets for new treatment to eradicate the disease. Core researchfacilities at several other academic medical centers will providespecialized support in analyzing data.

The SCOR officially began on October 1. "This grant from the Leukemia &Lymphoma Society creates a unique opportunity to produce advances against a very challenging form of leukemia," said Dr. Felix. "The SCOR grant bringstogether researchers from around the United States and harnesses ourefforts to build bridges between basic science and bedside treatments andhave the utmost impact for infants with this dread disease."

About The Children's Hospital of Philadelphia: The Children's Hospitalof Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited childrenworldwide. Its pediatric research program is among the largest in thecountry, ranking third in National Institutes of Health funding. Inaddition, its unique family-centered care and public service programs havebrought the 430-bed hospital recognition as a leading advocate for childrenand adolescents. For more information, visit http://www.chop.edu. Contact: John Ascenzi Phone: (267) 426-6055 Ascenzi@email.chop.edu